“For over a century, medicines have been developed almost exclusively for large populations. That’s changing.”
As science and technology change the therapeutic landscape, the RTLP is focused on establishing the appropriate pathway from patient diagnosis to access of an individualised medicine, mapping the responsibilities of stakeholders to each step, and identifying gaps in policy, regulation and legislation.
Our Goal
To put the system in place to ensure equitable and sustainable access to individualised medicines.
Patient diagnosis to treatment pathway
As technology moves us towards medicines for small cohorts of patients, innovative reimbursement frameworks are needed. Conventional measurements cannot be easily applied in individualised therapies due to extremely small patient numbers and consequent lack of clinical and cost-benefit evidence. Additionally, individualised medicines are currently considered research or fall under early access programmes which are not in most cases reimbursable. In a necessary shift toward utilising industry expertise and scalability, reimbursement will be critical to ensure equitable patient access
Reimbursement
Linking the patient to a therapeutic intervention early in the course of disease has proven to be crucial to arresting disease progression. As Genomics England and the NHS work together through routine care and research towards early genetic diagnosis, the RTLP will test the necessary infrastructure for rapidly coupling these newly diagnosed patients to treatment modalities appropriate for their condition.
Proportionate regulation
Genetic technologies have already shifted medicine from population-based therapeutics to stratified cohorts, and more recently this is further shifting to truly individualised medicines in the rare disease space on which the RTLP is focused. This necessitates a process transformation by regulators who are currently not positioned to appraise individualised medicines at scale today.
The RTLP aims to demonstrate a necessary shift toward proportionate regulation by exploring new approaches and learning from established practices and procedures, such as the surgery and stem cell transplant settings, where accredited centres of excellence allow for highly individualised risk assessment and consent between clinicians and parents/patients. A move from research to treatment in accredited centres, that work within pre-defined and agreed parameters based on prior knowledge and experience, represents a significant evolution in how we approve treatments for use within the health system.